In this condition, which can also be called “beta-thalassemia minor,” one of your two beta-globin genes is mutated. Researchers have identified more than 200 such mutations with a range of ...

Researchers reported Friday that a preclinical study may have discovered a gene therapy vector candidate for blood disorders like sickle cell disease (SCD) and beta-thalassemia that could avoid ...

Alpha-globin genes People have four alpha-globin genes located on chromosome 16. More than 130 possible changes to these genes are associated with alpha-thalassemia.

The functional beta-globin gene is added to a patient’s cells outside of the body (ex-vivo), and then infused into the patient. In September 2021.

Source: Hongeng S, et al. Abstract 1. Presented at: The 2021 TCT Meetings Digital Experience (virtual meeting); Feb. 8-12, 2021.

The delta globin gene sits upstream of the closely related beta globin gene in a gene cluster on chromosome 11. Attention turns to the delta globin gene on chromosome 11 as a potential key to ...

NEW ORLEANS —One-time treatment with the gene therapy betibeglogene autotemcel continued to demonstrate long-term efficacy among patients with transfusion-dependent beta-thalassemia, study ...

Understand the causes, disease processes and presenting symptoms of the inherited blood disorders, sickle cell and thalassaemia. This is a Self-assessment article and comes with a self-assessment test ...

Adult hemoglobin, expressed primarily after birth, contains four protein subunits—two beta-globin and two alpha-globin. Mutations in the beta-globin gene cause sickle cell disease and beta ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

In his approach, the beta-globin gene was transferred into the cells of two patients with beta-thalassamia. The experiment failed in part because the treated cells did not replicate. This incident led ...